The ‘valley of death’ in scientific parlance refers to the time period where biomedical discovery in the laboratory fails to result in a cure for a disease. While the last decade has seen extraordinary developments in regenerative biology, the translation to regenerative medicine will require successful negotiation of the ‘valley of death.’ In most cases, the scientific field has yet to prepare a model of stem cell manufacturing that will meet the Food and Drug Administration’s requirement of Good Manufacturing Practices and produce sufficient quantities of quality stem cells to meet patient need. This critical step requires the development of manufacturing technology and quality control standards for stem cell production. Using cardiac muscle cells as our model, this proposed project will develop these standards and practices to enable industry to meet regulatory requirements and produce safe and effective cures for heart disease. These lessons learned will be broadly applicable to quality production of stem cells for a broad range of pathological conditions which warrant a regenerative therapeutic option.
Protecting California’s investment in Regenerative Medicine includes prudent development of the techniques and methods required to meet regulatory requirements and the development of safe and effective cell-based therapeutics. The collapse of Geron Corp’s stem cell development program and the difficulties facing current stem cell manufacturers suggest that the lack of a defined set of Quality Control standards and methods for cardiac stem cell production are representative of a broader problem that could impede efforts to exploit bench discoveries, traverse the ‘valley of death,’ and industrialize the production of cellular therapeutics. In our proposed study, we will use stem cell-derived cardiac muscle cells as a model to develop protocols and standards for determining cell quality and their applicability for disease models in drug discovery and as cellular therapeutics in the clinical environment. The cardiac stem cell quality index we will develop benefits the State of California by facilitating safe and effective therapeutics for ailing Californians and enabling the California stem cell industry with the manufacturing practices required to meet regulatory requirements and achieve market dominance.